WOBURN, Mass.--(BUSINESS WIRE)--Jan 24, 2011 - ArQule, Inc.
(NASDAQ: ARQL) today announced the presentation of Phase 1 results
of a clinical trial among patients with metastatic colorectal
cancer (CRC) treated with ARQ 197, a selective small molecule
inhibitor of the c-MET receptor tyrosine kinase, in combination
with irinotecan and cetuximab. ARQ 197 is under development by
Daiichi Sankyo Co., Ltd. and ArQule.

Data presented at the ASCO 2011 Gastrointestinal Cancers
Symposium showed that this combination was well tolerated and
demonstrated encouraging anti-tumor activity in patients with
relapsed metastatic CRC. Among nine patients treated, one had a
complete response, two had partial responses and five had stable
disease. The systemic exposure of ARQ 197 with this combination
regimen was consistent with previous observations, and no
dose-limiting toxicities were observed.

“These results provide important support for the ongoing
Phase 2 randomized study of this combination,” said Dr. Brian
Schwartz, chief medical officer of ArQule. “We believe they
strengthen the rationale for this study, which includes the
previous demonstration of anti-cancer activity by ARQ 197 in a
human colorectal cancer xenograft model, as well as the link shown
between an increase in c-MET signaling and the development of
resistance to therapy with epidermal growth factor receptor (EGFR)
inhibitors.”

The ongoing Phase 2 study of ARQ 197 in CRC is enrolling
patients with the wild-type form of the KRAS gene who have received
front-line systemic therapy. The primary objective of the trial is
progression-free survival. Secondary objectives include overall
survival and objective response rate. Approximately 150 patients
will be enrolled at clinical trial sites in the U.S. and Europe.
The trial is being conducted by Daiichi Sankyo Pharma Development,
the global development arm of Daiichi Sankyo, the co-developer with
ArQule of ARQ 197 outside of certain countries in Asia.

Additional cancers currently being evaluated in randomized
trials of ARQ 197 as a single agent or in combination therapy
include non-small cell lung cancer (Phase 3) and hepatocellular
carcinoma (Phase 2). Patients, physicians and other healthcare
professionals seeking additional information regarding these and
other trials involving ARQ 197 may call 1-800-373-7827.

About ARQ 197 and c-MET

ARQ 197 is an orally available, selective inhibitor of c-Met, a
receptor tyrosine kinase that is currently in Phase 2 and Phase 3
clinical trials and is not yet approved for commercial sale. In
healthy adult cells, c-Met is present in normal levels to support
natural cellular function, but in cancer cells, c-Met is
inappropriately and continuously activated for unknown reasons.
When abnormally activated, c-Met plays multiple roles in aspects of
human cancer, including cancer cell growth, survival, angiogenesis,
invasion and metastasis.

Pre-clinical data have demonstrated that ARQ 197 inhibits c-Met
activation in a range of human tumor cell lines and shows
anti-tumor activity against several human tumor xenografts. In
clinical trials to date, treatment with ARQ 197 has been
well-tolerated and has resulted in tumor responses and prolonged
stable disease across a broad range of tumors.

In December 2008, ArQule and Daiichi Sankyo Co., Ltd. signed a
license, co-development and co-commercialization agreement to
co-develop ARQ 197 in the U.S., Europe, South America and the rest
of the world, excluding Japan, China (including Hong Kong), South
Korea and Taiwan, where Kyowa Hakko Kirin Co., Ltd. has exclusive
rights for development and commercialization.

About ArQule

ArQule is a biotechnology company engaged in the research and
development of next-generation, small-molecule cancer therapeutics.
The Company's targeted, broad-spectrum products and research
programs are focused on key biological processes that are central
to human cancers. ArQule's lead product, in Phase 2 and Phase 3
clinical development, is ARQ 197, an inhibitor of the c-Met
receptor tyrosine kinase. The Company has also initiated Phase 1
clinical testing with ARQ 621, designed to inhibit the Eg5 kinesin
motor protein, and with ARQ 736, designed to inhibit the RAF
kinases. ArQule's current discovery efforts, which are based on the
ArQule Kinase Inhibitor Platform (AKIP™), are focused on the
identification of novel kinase inhibitors that are potent,
selective and do not compete with ATP (adenosine triphosphate) for
binding to the kinase.

This press release contains forward-looking statements
regarding the progress of the Company's clinical trials, including
its Phase 2 trial with ARQ 197 in colorectal cancer and trials that
may be conducted by Daiichi Sankyo and/or Kyowa Hakko Kirin under
their agreements with the Company.These statements are
based on the Company's current beliefs and expectations, and are
subject to risks and uncertainties that could cause actual results
to differ materially.Positive information about early stage
clinical trial results is not necessarily indicative of clinical
efficacy and does not ensure that later stage or larger scale
clinical trials will be successful. For example, ARQ 197 may not
demonstrate promising therapeutic effect; in addition, this
compound may not demonstrate an appropriate safety profile in
further pre-clinical testing and in current, later stage or larger
scale clinical trials as a result of known or as yet unanticipated
side effects. The results achieved in later stage trials may not be
sufficient to meet applicable regulatory standards. Problems or
delays may arise during clinical trials or in the course of
developing, testing or manufacturing these compounds that could
lead the Company or its partner to discontinue development.Even if later stage clinical trials are successful, the risk
exists that unexpected concerns may arise from analysis of data or
from additional data or that obstacles may arise or issues be
identified in connection with review of clinical data with
regulatory authorities or that regulatory authorities may disagree
with the Company's view of the data or require additional data,
information or studies.In addition, the planned timing of
initiation and completion of clinical trials for ARQ 197 are
subject to the ability of the Company or Daiichi Sankyo, its
partner, and Kyowa Hakko Kirin, a licensee of ARQ 197, to enroll
patients, enter into agreements with clinical trial sites and
investigators, and other technical hurdles and issues that may not
be resolved.Moreover, Daiichi Sankyo has certain rights to
unilaterally terminate the ARQ 197 license, co-development and
co-commercialization agreement. Drug development involves a high
degree of risk. Only a small number of research and development
programs result in the commercialization of a product. Furthermore,
ArQule may not have the financial or human resources to pursue drug
discovery successfully in the future. For more detailed information
on the risks and uncertainties associated with the Company's drug
development and other activities see the Company's periodic reports
filed with the Securities and Exchange Commission. The Company does
not undertake any obligation to publicly update any forward-looking
statements.

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